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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the
Securities Exchange Act of 1934
Date of report (Date of earliest event reported):
June 16, 2026
Verastem,
Inc.
(Exact Name of Registrant as Specified in
Charter)
| Delaware |
|
001-35403 |
|
27-3269467 |
(State or Other Jurisdiction
of Incorporation) |
|
(Commission
File Number) |
|
(IRS Employer
Identification No.) |
| 117 Kendrick Street, Suite 500, Needham, MA |
|
02494 |
| (Address of Principal Executive Offices) |
|
(Zip Code) |
Registrant’s telephone number, including
area code: (781) 292-4200
(Former Name or Former Address, if Changed
Since Last Report)
Check the appropriate box below if the Form 8-K filing is intended
to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
| ¨ | Written communications pursuant to Rule 425 under
the Securities Act (17 CFR 230.425) |
| ¨ | Soliciting material pursuant to Rule 14a-12 under
the Exchange Act (17 CFR 240.14a-12) |
| ¨ | Pre-commencement communications pursuant to Rule 14d-2(b) under
the Exchange Act (17 CFR 240.14d-2(b)) |
| ¨ | Pre-commencement communications pursuant to Rule 13e-4(c) under
the Exchange Act (17 CFR 240.13e-4(c)) |
Securities registered pursuant to Section 12(b)
of the Act:
| Title of each class |
|
Trading
Symbol(s) |
|
Name
of each exchange on which registered |
| Common stock, $0.0001 par value per share |
|
VSTM |
|
The Nasdaq Capital Market |
Indicate by check mark whether the registrant is an emerging
growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities
Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging
growth company ¨
If
an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for
complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ¨
Item 7.01 Regulation FD Disclosure
On June 17, 2026, Verastem, Inc. (the “Company”)
issued a press release announcing positive updated results from RAMP 205 evaluating avutometinib plus defactinib in combination with standard-of-care
chemotherapy in first-line metastatic pancreatic ductal carcinoma (“mPDAC”).
A copy of this press release is furnished hereto as Exhibit 99.1
to this Current Report on Form 8-K.
Item 8.01 Other Events
Positive Updated Results from RAMP 205 Evaluating Avutometinib
Plus Defactinib in Combination with Standard-of-Care Chemotherapy in First-Line Metastatic Pancreatic Cancer
On June 17, 2026, the Company announced positive updated safety and
efficacy results from the RAMP 205 Phase 1b/2a trial Recommended Phase 2 Dose (“RP2D”) cohort of 29 patients evaluating avutometinib
plus defactinib in combination with gemcitabine and nab-paclitaxel in first-line mPDAC.
In the Phase 1b/2a trial, 29 patients were enrolled and treated at
the RP2D with avutometinib 2.4 mg twice weekly, defactinib 200 mg twice daily for 3 weeks on and one week off, and gemcitabine (800 mg/m2)
plus nab-paclitaxel (125 mg/m2) administered on Days 1, 8, and 15 of each 28-day cycle. At diagnosis, 90% of patients presented with metastatic
disease. As of the June 5, 2026 data cutoff, with a median follow up of 9.8 months, the combination demonstrated clinical activity, including an 86% overall survival rate at 6 months. The progression-free survival rate at six months was 68%, and the confirmed objective
response rate was 52%. At the RP2D dose level, the majority (83%) of patients experienced tumor shrinkage. Nine patients remain
on treatment at this dose level. Adverse events remained generally consistent with the previously reported safety and tolerability profile,
with no new safety signals observed.
Dosing of First Patient in TARGET-D 201 Phase 2 Registration-Directed
Trial of VS-7375
On June 16, 2026, the Company also announced
that the first patient has been dosed in the TARGET-D 201 Phase 2 registration-directed trial evaluating VS-7375, an investigational oral
KRAS G12D (ON/OFF) inhibitor, to treat patients with KRAS G12D-mutated mPDAC.
TARGET-D 201 is a Phase 2, open-label, multi-center study to evaluate
VS-7375 at 900 mg daily both as monotherapy and in combination with full-dose cetuximab in patients with second-line mPDAC.
Item
9.01. Financial Statements and Exhibits
| Exhibit No. |
|
Description |
| 99.1 |
|
Press Release, dated June 17, 2026 |
| 104 |
|
Cover Page Interactive Data File (embedded within the Inline XBRL document) |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934,
the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| |
VERASTEM, INC. |
| |
|
|
| Dated: June 17, 2026 |
By: |
/s/ Daniel W. Paterson |
| |
|
Daniel W. Paterson |
| |
|
President and Chief Executive
Officer |
Exhibit 99.1
Verastem Oncology Announces Positive Updated
Results from RAMP 205 Evaluating Avutometinib Plus Defactinib in Combination with Standard-of-Care Chemotherapy in First-Line Metastatic
Pancreatic Cancer
90% of patients in the study presented with
metastatic (Stage IV) disease at diagnosis
6-month overall survival rate was 86%; follow-up
continues and survival data continue to mature
BOSTON--(BUSINESS WIRE)—June 17, 2026--Verastem Oncology
(Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, today
announced positive updated safety and efficacy results from the RAMP 205 Phase 1b/2a Recommended Phase 2 Dose (RP2D) cohort of 29 patients
evaluating avutometinib plus defactinib in combination with gemcitabine and nab-paclitaxel in first-line metastatic pancreatic ductal
adenocarcinoma (PDAC).
KRAS is mutated in more than 90% of pancreatic cancers, making it a
key driver of tumor growth. The RAMP 205 trial was designed to evaluate whether simultaneous inhibition of KRAS-driven signaling and FAK-mediated
resistance pathways, in combination with standard-of-care chemotherapy, could improve outcomes for patients living with metastatic pancreatic
cancer.
“The updated data from the RAMP 205 trial provide important
clinical insights into the potential impact of combined RAF/MEK and FAK inhibition as a therapeutic option for pancreatic cancer.
While pancreatic cancer remains one of the most challenging cancers to treat, the early survival trends and deep
responses observed in this study indicate that avutometinib and defactinib are combinable with standard-of-care chemotherapy and
may help overcome resistance mechanisms inherent in pancreatic cancer and support further exploration of strategies designed to
address oncogenic signaling and mechanisms of treatment resistance,” said John Hayslip, M.D., chief medical officer at
Verastem Oncology. “We are grateful to PanCAN, the RAMP 205 investigators, and especially the patients and families who
participated in the trial.”
In the Phase 1b/2a study, 29 patients were enrolled and treated at the
RP2D with avutometinib 2.4 mg twice weekly (BIW), defactinib 200 mg twice daily (BID) for 3 weeks on and one week off, and
gemcitabine (800 mg/m2) plus nab-paclitaxel (125 mg/m2) administered on Days 1, 8, and 15 of each 28-day
cycle. At diagnosis, 90% of patients presented with metastatic disease. As of the June 5, 2026 data cutoff, with a median
follow up of 9.8 months, the combination demonstrated encouraging clinical activity, including an 86% overall survival (OS) rate at
6 months, with the OS data continuing to mature. The progression-free survival (PFS) rate at 6 months was 68%, and the confirmed
objective response rate (ORR) was 52% (15/29). At the RP2D dose, the majority (83%) of patients experienced tumor shrinkage. Nine
patients remain on treatment at this dose level. Adverse events remained generally consistent with the previously reported safety
and tolerability profile, with no new safety signals observed.
“For patients and their families facing a pancreatic cancer diagnosis,
every advance in research and understanding of the underlying biology driving this cancer matters,” said Anna Berkenblit, M.D.,
chief scientific and medical officer of the Pancreatic Cancer Action Network (PanCAN). “We awarded Verastem the PanCAN Therapeutic
Accelerator Award in 2022 to invest in research and development of novel treatment approaches. The results of the RAMP 205 trial underscore
the importance of continued research in the RAS/MAPK-pathway to help improve outcomes for patients living with pancreatic cancer.”
“We will continue to evaluate the potential role of avutometinib
plus defactinib in metastatic pancreatic cancer, including future development opportunities and potential strategic collaborations, informed
by the final overall survival results from the study as well as emerging data from VS-7375, our investigational potential best-in-class
oral KRAS G12D (ON/OFF) inhibitor, currently being evaluated in metastatic pancreatic cancer as both a monotherapy and in combination
regimens,” said Dan Paterson, president and chief executive officer of Verastem Oncology. “We will also assess opportunities
to share these data in the future, including at a medical meeting”.
In May 2022, Verastem Oncology was selected by PanCAN
to receive the inaugural PanCAN Therapeutic Accelerator Award, supporting evaluation of avutometinib in combination with defactinib in
front-line metastatic pancreatic cancer. Designed to accelerate the development of new pancreatic cancer treatments, the award provided
Verastem with $3.8M following a rigorous, competitive process involving scientific, business, and programmatic review from leading
experts in the field. In parallel, a working group led by PanCAN was formed as a partnership between Verastem and the academic community
to further understand the science behind and the potential of this investigational treatment combination to improve outcomes for patients.
About Metastatic Pancreatic Cancer
Pancreatic cancer is the third leading cause of cancer-related death
in the U.S. and seventh leading cause of cancer-associated mortality worldwide. Metastatic pancreatic cancer, or stage IV disease,
occurs when the cancer spreads beyond the pancreas to distant organs. More than 90% of pancreatic cancers harbor KRAS mutations, underscoring
the central role of KRAS in the development and the progression of the disease. Approximately 40% of pancreatic tumors harbor a KRAS G12D
mutation, the most prevalent subtype in pancreatic cancer. Patients with KRAS G12D-mutant tumors often have poorer outcomes, underscoring the
need for therapies designed specifically to inhibit this mutation potently and for a long duration. Each year, more than
30,000 people in the U.S. and over 240,000 people globally are diagnosed with metastatic pancreatic cancer. There has been minimal progress
with treatment, and the five-year survival rate remains approximately 3%. Current treatment approaches may include surgery, chemotherapy,
radiation therapy, targeted therapies, or a combination of these modalities.
About RAMP 205 Phase 1b/2a Study
RAMP 205 is a multicenter, open-label, single arm Phase 1b/2a study to evaluate the safety, tolerability, and efficacy of avutometinib and defactinib in combination with standard of care chemotherapy
(gemcitabine and nab-paclitaxel) in patients with previously untreated metastatic pancreatic ductal adenocarcinoma. Part A of the
study evaluated varied dose and schedule combinations to determine the recommended Phase 2 dose for expansion into Part B. RAMP 205
is supported by a PanCAN Therapeutic Accelerator Award.
About AVMAPKI and FAKZYNJA Combination Therapy
AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking
the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking
RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib
and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance
of RAS/MAPK pathway-dependent tumors.
The U.S. Food and Drug Administration (FDA) approved AVMAPKI® FAKZYNJA® CO-PACK
(avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior
systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description
of clinical benefit in a confirmatory trial. Verastem is conducting RAMP 301 (GOG-3097/ENGOT-ov81/GTG-UK) (NCT06072781), an
international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal
therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC) with and without a KRAS mutation. Verastem is
also evaluating avutometinib plus defactinib with standard-of-care chemotherapy as a potential treatment in the first-line for patients
with advanced pancreatic cancer (RAMP 205; NCT05669482). Avutometinib and defactinib are not approved by the FDA or any other regulatory
authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are
approved by the FDA or any other regulatory authority on a stand-alone basis for any use.
AVMAPKI FAKZYNJA CO-PACK U.S. Indication
Indication
AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients
with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.
This indication is approved under accelerated approval based on tumor
response rate and duration of response. Continued approval for this indication may be contingent upon verification and description
of clinical benefit in a confirmatory trial.
Important Safety Information
Warnings and Precautions
| · | Ocular Toxicities: Ocular toxicities, including visual impairment
and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter,
and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently
discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity. |
| · | Serious Skin Toxicities: Skin toxicities, including photosensitivity
and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt,
reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration. |
| · | Hepatotoxicity: Monitor liver function tests prior to each cycle,
on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity
and persistence of abnormality. |
| · | Rhabdomyolysis: Monitor creatine phosphokinase prior to the start
of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis
or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction. |
| · | Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal
harm. Advise patients of the potential risk to a fetus and to use effective contraception. |
Adverse Reactions
The most common (≥ 25%) adverse reactions, including laboratory
abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal
pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased
lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis,
pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased
neutrophil count.
Drug Interactions
| · | Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with
AVMAPKI FAKZYNJA CO-PACK. |
| · | Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI
FAKZYNJA CO-PACK. |
| · | Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with
warfarin and use an alternative to warfarin. |
| · | Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA
CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer
FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid. |
Use in Specific Populations
| · | Lactation: Advise not to breastfeed. |
| · | Fertility: May impair fertility in males and females. |
Click here for full Prescribing Information.
About Verastem Oncology
Verastem Oncology (Nasdaq: VSTM) is a biopharmaceutical company committed
to developing and commercializing new medicines to improve the lives of patients diagnosed with RAS/MAPK pathway-driven cancers. Verastem markets
AVMAPKI® FAKZYNJA® CO-PACK in the U.S. Our pipeline is focused on novel small molecule
drugs that inhibit critical signaling pathways in cancer that promote cancer cell survival and tumor growth, including RAF/MEK inhibition,
FAK inhibition, and KRAS G12D inhibition. For more information, please visit www.verastem.com and follow us on LinkedIn.
Forward-Looking Statements
This press release includes forward-looking statements. These forward-looking
statements generally can be identified by the use of words such as “anticipate,” “expect,” “plan,”
“could,” “may,” “believe,” “estimate,” “forecast,” “goal,” “project,”
and other words of similar meaning. Such forward-looking statements address various matters about, among other things, Verastem Oncology’s
programs and product candidates, strategy, future plans and prospects, including statements related to the potential for and timing of
commercialization of product candidates, the expected outcome and benefits of the Company’s collaboration with GenFleet Therapeutics
(Shanghai), Inc., the timing of commencing and completing trials and compiling data, the expected timing of the presentation of data
by the Company and the potential clinical value of various of the Company’s clinical trials. Each forward-looking statement contained
in this press release is subject to risks and uncertainties that could cause actual results to differ materially from those expressed
or implied by such statement. Applicable risks and uncertainties include, among others: the uncertainties inherent in research and development,
such as the possibility of negative or unexpected results of clinical trials; that we may not see a return on investment on the payments
we have and may continue to make pursuant to the collaboration and option agreement with GenFleet, or that GenFleet may fail to fully
perform under the agreement; that we may not be successful in our continued commercialization of AVMAPKI FAKZYNJA CO-PACK; that the development
and commercialization of our product candidates may take longer or cost more than planned, including as a result of conducting additional
studies or our decisions regarding execution of such commercialization; that data may not be available when expected; risks associated
with preliminary and interim data, which may not be representative of more mature data; risks associated with the recent changes in administration
policy or actions that may create regulatory uncertainty that may adversely affect our business; risks associated with the current administration’s
reductions to the FDA’s workforce and any subsequent reductions that may lead to disruptions and delays in the FDA’s review
and oversight of our product candidates and impact the FDA’s ability to provide timely feedback on our development programs; that
our product candidates may not receive regulatory approval, become commercially successful products, or result in new treatment options
being offered to patients; and the risks identified under the heading "Risk Factors" as detailed in the Company’s Annual
Report on Form 10-K for the year ended December 31, 2025, as filed with the Securities and Exchange Commission (SEC) on March 4,
2026, as well as the other information we file with the SEC, are possibly realized. We caution investors not to place considerable
reliance on the forward-looking statements contained in this press release. You are encouraged to read our filings with the SEC,
available at www.sec.gov, for a discussion of these and other risks and uncertainties. The forward-looking statements in this press
release speak only as of the date of this press release, and we undertake no obligation to update or revise any of these statements. Our
business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others
should give careful consideration to these risks and uncertainties.
For Investor and Media Inquiries:
Julissa Viana
Senior Vice President, Corporate Communications
Investor Relations and Patient Advocacy
investors@verastem.com or
media@verastem.com
